A truly thankful moment

This year, as my family celebrates one of our favorite holidays—as we take turns chasing my 2 year-old granddaughter around the house, as I try yet again to time the turkey and the stuffing and the sweet potatoes to all be done at least around the same time, as the crowds from football games cheer in the background—we get to do something we have never done before. We get to be grateful for the first drug approval in the U.S. for Duchenne.

For years, decades now, I have worked towards, hoped for, and prayed for this moment. This Thanksgiving, it is here: an FDA approval.

Yes, this therapy, EXONDYS 51™ (aka eteplirsen), is limited to 13% of the Duchenne population. Yes, the FDA has required further clinical trials from Sarepta and the decision has been fraught with controversy. Yes, we are just starting to see the challenges around access and coverage we will be facing in our community.

But you know what? Today, we are 13% closer to identifying effective therapies for every family. And this community stands ready for the other challenges the path to full approval will entail.

And it sure beats where we were this time last year. And the year before. And the year before.

We're so thankful for this community!

We’re hopeful that this approval will lead to more positive results for all people with Duchenne. To make sure we were ready for this moment, we spent years laying the groundwork for the critical conversations that must now follow. Many of you have participated in our efforts in Washington, D.C. by attending our Annual Advocacy Conference and by writing or calling your members of Congress. Thanks to your help, we’re now ready to fight for access and coverage. Important conversations are underway and we will keep you updated and informed about how you can help.

Meanwhile, we continue to certify the best clinics in the country, and I’m thankful that so many of our children now receive the care they deserve. Good care also means constantly revisiting and revamping how we define good. It means convening experts throughout the year and prescribing recommendations to care providers so that when your son or daughter has an appointment, you can be confident that they are receiving pertinent information.

To address the needs of 100% of the Duchenne community, PPMD has always and will always invest in a wide array of potential therapies, from early-stage research to late-stage clinical trials. The key to our research strategy is innovation in potentially game-changing solutions like gene therapy and CRISPR/Cas9. We have said for years that it will take a combination of therapies to treat Duchenne, and we want to make sure we have a full pipeline of therapies that can work together to tackle the entire progression of the disease.

Our community is being watched by the world. The battles we are fighting in the war to end Duchenne are being—and will continue to be—closely examined by every stakeholder in the rare disease space. This is a moment—a moment of extraordinary opportunity for our community. And I am grateful that we are finally here.

None of this is to say that we are done. In fact, we are just getting started.

Thanksgiving is a time to take pause. The turkey may come out a little dry. The 2-year-old may end up taking a black Sharpie to your wall. Your football team may fumble, and may even lose the game.

What is important about today, though—and every day really—is taking a moment to be grateful for the victories. Take a deep breath. Celebrate those you love and those who love you. Remember what you have.

You—the incredible person reading this note whose life has in some way been affected by Duchenne—you are who I am thankful for this Thanksgiving. You share this journey with me and the entire PPMD family. And for that I am truly grateful.

I wish you and your loved ones a wonderful Thanksgiving. Remember that PPMD continues to stand by your side as we get closer and closer to the day we end Duchenne.